Prophilaxis of Angioedema Attacks on Patients with Hereditary Angioedema (HAE). MedDRA version: 23.1 Level: PT Classification code 10019860 Term: Hereditary angioedema System Organ Class: 10010331 - Congenital, familial and genetic disorders MedDRA version: 20.0 Level: HLGT Classification code 10027664 Term: Congenital and hereditary disorders NEC System Organ Class: 10010331 - Congenital, familial and genetic disorders
Conditions
Interventions
Trade Name: RUCONEST - 2100 U - POLVERE PER SOLUZIONE INIETTABILE - USO ENDOVENOSO - FLACONCINO(VETRO) 1 FLACONCINO DA 2100 U
Product Name: Ruconest
Product Code: rhC1INH
Pharmaceutical Form: Powder f
Sponsors
PHARMING GROUP NV
Eligibility
Sex/Gender
All
Inclusion criteria
Inclusion criteria: 1. Males or non-pregnant females, 13 years of age or older 2. Laboratory confirmed diagnosis of HAE (functional C1INH level =65 years) yes F.1.3.1 Number of subjects for this age range 5
Exclusion criteria
Exclusion criteria: 1. Patients with medical history of allergy to rabbits or rabbit-derived products (including rhC1INH) 2. Diagnosis of acquired angioedema (AAE) including positive anti-C1INH antibodies with a low C1q level and/or clinical features suggestive of AAE 3. Patients who are pregnant, or breastfeeding, or are currently intending to become pregnant 4. Treatment with any investigational drug in the past 30 days 5. Patients with any condition or treatment that, in the opinion of the Investigator, might interfere with the evaluation of study objectives 6. Patients currently treated with angiotensin-converting enzyme (ACE) inhibitors 7. Patients who are using pdC1INH for prophylaxis or acute treatment of HAE attacks will need to discontinue therapy for at least 7 days prior to their first visit of Period 1. Patients may treat any HAE attacks between Screening and Randomization with open-label rhC1INH, HAE-specific medications (icatibant, ecallantide), or other symptomatic medications (narcotics, anti-emetics) as required.
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Main Objective: To evaluate the efficacy of rhC1INH in the prophylaxis of angioedema attacks in patients with HAE.;Secondary Objective: To evaluate the safety of rhC1INH in the prophylaxis of angioedema attacks in patients with HAE.;Primary end point(s): The primary endpoint is the number of HAE attacks during each 28 days treatment period.;Timepoint(s) of evaluation of this end point: All endpoints will be evaluated in each study period. | — |
Secondary
| Measure | Time frame |
|---|---|
| Secondary end point(s): The secondary endpoint the proportion of subjects who experience a clinical response, defined as a = 50% reduction in the number of attacks from the placebo treatment period to the active study drug treatment period.;Timepoint(s) of evaluation of this end point: All endpoints will be evaluated in each study period. | — |
Countries
Canada, Czechia, Czech Republic, Israel, Italy, North Macedonia, Poland, Romania, Serbia, United States
Contacts
Public ContactClinical Development Department
Pharming Group NV
Outcome results
None listed